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Quick Info
Currently Recruiting
Trial Type
Treatment Type
Drug Trials
Not Available
Start Date
Transposon Therapeutics, Inc.
Contact Information
    Contact information unknown.
Belgium, Other
VIB-KU Leuven Center for Brain & Disease Research, Leuven, 3000, Belgium
Contact: Philip Van Damme, MD, PhD
France, Other
CHU Lille - CMRR Hôpital Roger Salengro, Lille, 59037, France
Contact: Florence Pasquier, MD, PhD
CHU Dupuytren, Limoges, Limoges, 87042, France
Contact: Philippe Couratier, MD, PhD
Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux, Paris, 75013, France
Contact: Richard Levy, MD, PhD
Germany, Other
Universitaetsklinikum Ulm - Klinik fuer Neurologie, Ulm, 89081, Germany
Contact: Albert C Ludolph, MD
Spain, Other
Hospital de la Santa Creu i Sant Pau, Barcelona, 08025, Spain
Contact: Alberto Lleo Bisa, MD, PhD
Hospital Universitario Vall d'Hebron, Barcelona, 08035ES, Spain
Contact: Raul Juntas Morales, MD
Complejo Hospitalario Universitario de Santiago (CHUS), Santiago de Compostela, 15706ES, Spain
Contact: Julio Pardo Fernandez, MD, PhD
Hospital Universitari I Politècnic La Fe, Valencia, 46026, Spain
Contact: Miquel Baquero Toledo, MD
United States, California
University of California San Diego, La Jolla, CA, 92037, United States
Contact: Dominic Ferrey, MD
University of California Irvine - ALS & Neuromuscular Center, Orange, CA, 92868, United States
Contact: Namita A Goyal, MD
University of California at San Francisco/UCSF, San Francisco, CA, 55455, United States
Contact: Adam Boxer, MD, PhD
United States, Maryland
John Hopkins University, Baltimore, MD, 21287, United States
Contact: Chiadi Onyike, M.B.B.S., MD
Johns Hopkins Outpatient Center, Baltimore, MD, 21287, United States
Contact: Jeffrey D Rothstein, MD, PhD
United States, Massachusetts
Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic, Boston, MA, 02114, United States
Contact: Doreen Ho, MD
United States, Minnesota
Mayo Family Clinic Northwest, Rochester, MN, 55905, United States
Contact: Bradley Boeve, MD
United States, New York
Hospital for Special Surgery, New York, NY, 10021, United States
Contact: Dale J Lange, MD
Columbia University Medical Center - The Neurological Institute of New York, New York, NY, 10032, United States
Contact: Lawrence Honig, MD, PhD
United States, North Carolina
The University of North Carolina at Chapel Hill, Department of Neurology, Chapel Hill, NC, 27599, United States
Contact: Andrea Bozoki, MD
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
>60% (SVC)
Months Since Onset
Number of months since first symptoms of ALS.
<3 Years
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Open Label
Update Notes
Site removed
Sites added, protocol updates
Sites added
Sites added
Study recruiting
Outcome measures updates

Other Information

This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).
Inclusion Criteria:
- Documentation of a clinical genetic test demonstrating a hexanucleotide repeat expansion (HRE) in the C9orf72 gene - Has a reliable caregiver/informant to accompany the patient to all study visits For patients with ALS (with or without FTD):
- Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria - Onset of weakness within 3 years prior to Screening - Slow vital capacity (SVC) ≥ 60% of predicted normal adjusted for sex, age, and height (from the sitting position) - Able to perform reproducible pulmonary function tests.
- ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 and score of 3 or 4 on Item #3 (swallowing) at Screening For patients with FTD:
- A gradual, progressive decline in behavior, language, or motor function consistent with mild cognitive impairment, mild behavioral impairment, mild cognitive/behavioral impairment, behavioral variant FTD, primary progressive aphasia, or amnestic syndrome - CDR Dementia Staging Instrument plus National Alzheimer's Coordinating Center Behavior and Language Domains (CDR plus NACC FTLD) global score of 0.5-2.0 at Screening Exclusion Criteria:
- Presence of other significant neurological or psychiatric disorders - History of clinically significant brain abnormality - Clinically significant medical illness - Tracheostomy or diaphragmatic pacing - Autoimmune disease requiring treatment or management (quiescent rheumatoid arthritis, psoriasis, or controlled Type 1 diabetes are acceptable) - History of human immunodeficiency virus (HIV) or hepatitis B infection, or any active infection during Screening, unless the patient will have been symptom-free for at least 30 days prior to randomization
This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 2-arm study with a long-term, open-label treatment phase in patients with C9ORF72 ALS and/or FTD. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open-label Treatment Period, and a Follow-up Visit 4 weeks post-treatment.
  • Transposon Therapeutics, Inc.
Trial Protocol as Published on
NCT04993755 (First Published: 7/19/2021)